CA2946309C - Synthetic bcl11a micrornas for treating hemoglobinopathies - Google Patents

Synthetic bcl11a micrornas for treating hemoglobinopathies Download PDF

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CA2946309C
CA2946309C CA2946309A CA2946309A CA2946309C CA 2946309 C CA2946309 C CA 2946309C CA 2946309 A CA2946309 A CA 2946309A CA 2946309 A CA2946309 A CA 2946309A CA 2946309 C CA2946309 C CA 2946309C
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cells
cell
bcl11a
seq
nucleic acid
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CA2946309A1 (en
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Michael MILSOM
David A. Williams
Richard Gregory
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Boston Childrens Hospital
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Boston Childrens Hospital
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1135—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against oncogenes or tumor suppressor genes
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/141—MicroRNAs, miRNAs
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16032—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
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  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Analytical Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2946309A 2014-04-25 2015-04-24 Synthetic bcl11a micrornas for treating hemoglobinopathies Active CA2946309C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461984247P 2014-04-25 2014-04-25
US61/984,247 2014-04-25
US201462066783P 2014-10-21 2014-10-21
US62/066,783 2014-10-21
PCT/US2015/027527 WO2015164750A2 (en) 2014-04-25 2015-04-24 Compositions and methods to treating hemoglobinopathies

Publications (2)

Publication Number Publication Date
CA2946309A1 CA2946309A1 (en) 2015-10-29
CA2946309C true CA2946309C (en) 2021-11-09

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CA2946309A Active CA2946309C (en) 2014-04-25 2015-04-24 Synthetic bcl11a micrornas for treating hemoglobinopathies

Country Status (17)

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US (4) US10287588B2 (es)
EP (2) EP3134130B1 (es)
JP (2) JP6514717B2 (es)
KR (1) KR102390629B1 (es)
CN (2) CN112852812A (es)
AU (1) AU2015249381B2 (es)
BR (1) BR112016024565A2 (es)
CA (1) CA2946309C (es)
DK (1) DK3134130T3 (es)
ES (1) ES2744186T3 (es)
IL (1) IL248469B (es)
MX (1) MX375592B (es)
RU (1) RU2707540C2 (es)
SA (1) SA516380143B1 (es)
SG (2) SG10201809290SA (es)
WO (1) WO2015164750A2 (es)
ZA (1) ZA201607540B (es)

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